CALL US NOW (833) 525-3266


Orchard Therapeutics’ OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy

Orchard’s fourth Rare Pediatric Disease Designation for autologous ex vivo gene therapy. Boston, USA and London, UK, May 03, 2018 / B3C newswire / — Orchard Therapeutics, a leading commercial stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies, today announced that the

Read More

NORD Statement on Governor Bevin’s Veto of Kentucky Senate Bill 7

Washington, D.C., April 11, 2018 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, released the following statement regarding Governor Matt Bevin’s Veto of SB 7, the Kentucky Rare Disease Advisory Council: “Last week, Kentucky Governor Matt Bevin

Read More


Progress Report: Dr. Florian Eichler’s AMN Gene Therapy Pilot Study

Last year, The Myelin Project (in partnership with the Cure ALD Foundation) granted $50,000 to Dr. Florian Eichler’s pilot study of gene therapy in adrenomyeloneuropathy (AMN). Dr. Eichler and his team established an AMN animal model proof of concept showing a gene therapy approach could provide benefit where no treatment

Read More

Making Miracles Possible for Kids with Leukodystrophy

In 2013, a year after Pat and Maria Carr learned that their youngest child had one of the 50 forms of leukodystrophy, a degenerative, genetic neurological condition, they knew they wanted to help. It was too late to save Cal, but the Carrs were determined to give children with leukodystrophy

Read More

bluebird bio Publishes Interim Data from Starbeam Study of Lenti-DTM Drug in Cerebral Adrenoleukodystrophy (CALD) Patients

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Oct. 4, 2017– bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for serious genetic diseases and T cell-based immunotherapies for cancer, today announced that interim data from an initial cohort of 17 patients in the ongoing Phase 2/3 Starbeam Study (ALD-102)

Read More


News, events, and more...